JP2021533803A - Fasl免疫モジュレート遺伝子治療組成物および使用方法 - Google Patents

Fasl免疫モジュレート遺伝子治療組成物および使用方法 Download PDF

Info

Publication number
JP2021533803A
JP2021533803A JP2021509984A JP2021509984A JP2021533803A JP 2021533803 A JP2021533803 A JP 2021533803A JP 2021509984 A JP2021509984 A JP 2021509984A JP 2021509984 A JP2021509984 A JP 2021509984A JP 2021533803 A JP2021533803 A JP 2021533803A
Authority
JP
Japan
Prior art keywords
sequence
rna
present disclosure
promoter
protein
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
JP2021509984A
Other languages
English (en)
Japanese (ja)
Inventor
デイビッド エー. ネルズ,
Original Assignee
ロックアネイビオ, インコーポレイテッド
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ロックアネイビオ, インコーポレイテッド filed Critical ロックアネイビオ, インコーポレイテッド
Publication of JP2021533803A publication Critical patent/JP2021533803A/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/0005Vertebrate antigens
    • A61K39/001Preparations to induce tolerance to non-self, e.g. prior to transplantation
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70575NGF/TNF-superfamily, e.g. CD70, CD95L, CD153, CD154
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases RNAses, DNAses
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Transplantation (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
JP2021509984A 2018-08-24 2019-08-26 Fasl免疫モジュレート遺伝子治療組成物および使用方法 Pending JP2021533803A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862722550P 2018-08-24 2018-08-24
US62/722,550 2018-08-24
PCT/US2019/048148 WO2020041791A1 (fr) 2018-08-24 2019-08-26 Compositions de thérapie génique immunomodulatrice fasl et procédés d'utilisation

Publications (1)

Publication Number Publication Date
JP2021533803A true JP2021533803A (ja) 2021-12-09

Family

ID=69591397

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021509984A Pending JP2021533803A (ja) 2018-08-24 2019-08-26 Fasl免疫モジュレート遺伝子治療組成物および使用方法

Country Status (6)

Country Link
US (1) US20220175960A1 (fr)
EP (1) EP3841116A4 (fr)
JP (1) JP2021533803A (fr)
AU (1) AU2019326617A1 (fr)
CA (1) CA3110282A1 (fr)
WO (1) WO2020041791A1 (fr)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3802812A4 (fr) * 2018-06-08 2022-03-30 Locanabio, Inc. Compositions de protéines de fusion ciblant l'arn et méthodes d'utilisation
WO2021195025A1 (fr) * 2020-03-25 2021-09-30 City Of Hope Arn antisens pour le traitement du coronavirus associé au sras
WO2023004365A1 (fr) * 2021-07-20 2023-01-26 Sio Gene Therapies Inc. Constructions de vecteurs pour l'administration d'acides nucléiques codant pour des sous-unités complexes d'activateurs de protéasome thérapeutiques et leurs procédés d'utilisation
AU2022377400A1 (en) * 2021-11-01 2024-05-02 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing psd3 expression
WO2023242469A1 (fr) * 2022-06-13 2023-12-21 Turun Yliopisto Oligonucléotides pour moduler l'immunosuppression médiée par les lymphocytes t régulateurs

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6544523B1 (en) * 1996-11-13 2003-04-08 Chiron Corporation Mutant forms of Fas ligand and uses thereof
WO1998052615A1 (fr) * 1997-05-22 1998-11-26 Uab Research Foundation Regulation de la reponse immunitaire a des antigenes specifiques
US6451759B1 (en) * 1998-01-14 2002-09-17 The Regents Of The University Of California Noncleavable Fas ligand
WO2002072798A1 (fr) * 2001-03-13 2002-09-19 Johns Hopkins University School Of Medicine Cellules hematopoietiques exprimant fas ligand, destinees a une transplantation
US20030211509A1 (en) * 2002-03-26 2003-11-13 Wiley Steven R. TNF-delta ligand and uses thereof
WO2003092723A1 (fr) * 2002-05-03 2003-11-13 Nikolai Rainov Utilisation de fasl dans le traitement de maladies neurodegeneratives
GB201507104D0 (en) * 2015-04-27 2015-06-10 Ucl Business Plc Nucleic acid construct
JP7012645B2 (ja) * 2016-01-11 2022-01-28 ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー キメラタンパク質および免疫治療の方法

Also Published As

Publication number Publication date
AU2019326617A1 (en) 2021-03-18
CA3110282A1 (fr) 2020-02-27
US20220175960A1 (en) 2022-06-09
EP3841116A1 (fr) 2021-06-30
WO2020041791A1 (fr) 2020-02-27
EP3841116A4 (fr) 2022-05-25

Similar Documents

Publication Publication Date Title
JP2021526858A (ja) Rna標的化融合タンパク質組成物および使用方法
EP3289080B1 (fr) Thérapie génique pour maladies autosomiques dominantes
JP2021533803A (ja) Fasl免疫モジュレート遺伝子治療組成物および使用方法
US20220127621A1 (en) Fusion proteins and fusion ribonucleic acids for tracking and manipulating cellular rna
JP2021526860A (ja) 適応免疫をモジュレートするための組成物および方法
JP2018500391A (ja) 対象とする細胞を選択的に除去する方法及び組成物
US20210009987A1 (en) Rna-targeting knockdown and replacement compositions and methods for use
CN110214149B (zh) 用于治疗疼痛相关病症的材料和方法
JP2023551873A (ja) Cagリピート病を処置するためのrna標的化組成物および方法
JP2023551874A (ja) 筋強直性ジストロフィー1型を処置するためのrna標的化組成物および方法
JP2023517326A (ja) フォークヘッドボックスp3(foxp3)遺伝子発現をモジュレートするための組成物および方法
CN116801901A (zh) 用于治疗1型强直性肌营养不良的靶向rna的组合物和方法
KR20210151110A (ko) Scn9a 또는 scn10a 유전자를 변형시키기 위한 유전자-편집 시스템 및 이의 방법 및 용도
CN117320741A (zh) 用于治疗cag重复疾病的靶向rna的组合物和方法

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20220824

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20230728

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20240301